JP2019516680A5 - - Google Patents
Download PDFInfo
- Publication number
- JP2019516680A5 JP2019516680A5 JP2018557087A JP2018557087A JP2019516680A5 JP 2019516680 A5 JP2019516680 A5 JP 2019516680A5 JP 2018557087 A JP2018557087 A JP 2018557087A JP 2018557087 A JP2018557087 A JP 2018557087A JP 2019516680 A5 JP2019516680 A5 JP 2019516680A5
- Authority
- JP
- Japan
- Prior art keywords
- oligonucleotide
- pharmaceutically acceptable
- smg
- composition
- acceptable salt
- Prior art date
- Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
- Pending
Links
- 108091034117 Oligonucleotide Proteins 0.000 claims 40
- 239000000203 mixture Substances 0.000 claims 13
- 150000003839 salts Chemical class 0.000 claims 9
- 239000008194 pharmaceutical composition Substances 0.000 claims 8
- 159000000000 sodium salts Chemical group 0.000 claims 7
- 238000000034 method Methods 0.000 claims 6
- JLCPHMBAVCMARE-UHFFFAOYSA-N [3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[3-[[3-[[3-[[3-[[3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-[[5-(2-amino-6-oxo-1H-purin-9-yl)-3-hydroxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxyoxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(5-methyl-2,4-dioxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(6-aminopurin-9-yl)oxolan-2-yl]methoxy-hydroxyphosphoryl]oxy-5-(4-amino-2-oxopyrimidin-1-yl)oxolan-2-yl]methyl [5-(6-aminopurin-9-yl)-2-(hydroxymethyl)oxolan-3-yl] hydrogen phosphate Polymers Cc1cn(C2CC(OP(O)(=O)OCC3OC(CC3OP(O)(=O)OCC3OC(CC3O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c3nc(N)[nH]c4=O)C(COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3COP(O)(=O)OC3CC(OC3CO)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3ccc(N)nc3=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cc(C)c(=O)[nH]c3=O)n3cc(C)c(=O)[nH]c3=O)n3ccc(N)nc3=O)n3cc(C)c(=O)[nH]c3=O)n3cnc4c3nc(N)[nH]c4=O)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)n3cnc4c(N)ncnc34)O2)c(=O)[nH]c1=O JLCPHMBAVCMARE-UHFFFAOYSA-N 0.000 claims 4
- 125000000956 methoxy group Chemical group [H]C([H])([H])O* 0.000 claims 4
- 238000012986 modification Methods 0.000 claims 4
- 230000004048 modification Effects 0.000 claims 4
- RYYWUUFWQRZTIU-UHFFFAOYSA-K thiophosphate Chemical group [O-]P([O-])([O-])=S RYYWUUFWQRZTIU-UHFFFAOYSA-K 0.000 claims 4
- 229910019142 PO4 Chemical group 0.000 claims 3
- 108020004707 nucleic acids Proteins 0.000 claims 3
- 150000007523 nucleic acids Chemical class 0.000 claims 3
- 102000039446 nucleic acids Human genes 0.000 claims 3
- 239000010452 phosphate Chemical group 0.000 claims 3
- 229910001415 sodium ion Inorganic materials 0.000 claims 3
- 208000023105 Huntington disease Diseases 0.000 claims 2
- 229920002521 macromolecule Polymers 0.000 claims 2
- 108700028369 Alleles Proteins 0.000 claims 1
- 238000003776 cleavage reaction Methods 0.000 claims 1
- 230000000295 complement effect Effects 0.000 claims 1
- 239000003085 diluting agent Substances 0.000 claims 1
- 239000003937 drug carrier Substances 0.000 claims 1
- 239000004615 ingredient Substances 0.000 claims 1
- 239000000546 pharmaceutical excipient Substances 0.000 claims 1
- 229920000642 polymer Polymers 0.000 claims 1
- 230000007017 scission Effects 0.000 claims 1
Applications Claiming Priority (7)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| US201662331960P | 2016-05-04 | 2016-05-04 | |
| US62/331,960 | 2016-05-04 | ||
| USPCT/US2016/043542 | 2016-07-22 | ||
| PCT/US2016/043542 WO2017015555A1 (en) | 2015-07-22 | 2016-07-22 | Oligonucleotide compositions and methods thereof |
| US201762447832P | 2017-01-18 | 2017-01-18 | |
| US62/447,832 | 2017-01-18 | ||
| PCT/US2017/030753 WO2017192664A1 (en) | 2016-05-04 | 2017-05-03 | Oligonucleotide compositions and methods thereof |
Publications (2)
| Publication Number | Publication Date |
|---|---|
| JP2019516680A JP2019516680A (ja) | 2019-06-20 |
| JP2019516680A5 true JP2019516680A5 (enExample) | 2020-06-18 |
Family
ID=60203309
Family Applications (1)
| Application Number | Title | Priority Date | Filing Date |
|---|---|---|---|
| JP2018557087A Pending JP2019516680A (ja) | 2016-05-04 | 2017-05-03 | オリゴヌクレオチド組成物およびその方法 |
Country Status (6)
| Country | Link |
|---|---|
| US (2) | US10724035B2 (enExample) |
| EP (1) | EP3452596A4 (enExample) |
| JP (1) | JP2019516680A (enExample) |
| CN (1) | CN109477108A (enExample) |
| MA (1) | MA45270A (enExample) |
| WO (1) | WO2017192664A1 (enExample) |
Families Citing this family (81)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| CN112007045B (zh) | 2012-07-13 | 2026-01-06 | 波涛生命科学有限公司 | 手性控制 |
| DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| ES2810375T3 (es) | 2014-12-17 | 2021-03-08 | Proqr Therapeutics Ii Bv | Edición direccionada de ARN |
| WO2017015575A1 (en) | 2015-07-22 | 2017-01-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3359523A4 (en) | 2015-10-09 | 2019-07-24 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR |
| KR20220100719A (ko) | 2015-12-10 | 2022-07-15 | 피티씨 테라퓨틱스, 인크. | 헌팅턴병 치료 또는 개선을 위한 조성물 |
| AU2017234150B2 (en) | 2016-03-13 | 2021-09-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| JP2019516680A (ja) | 2016-05-04 | 2019-06-20 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物およびその方法 |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| EP3475424A1 (en) | 2016-06-22 | 2019-05-01 | ProQR Therapeutics II B.V. | Single-stranded rna-editing oligonucleotides |
| CA3035293A1 (en) | 2016-09-01 | 2018-03-08 | Proqr Therapeutics Ii B.V. | Chemically modified single-stranded rna-editing oligonucleotides |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| CN117298290A (zh) | 2016-12-19 | 2023-12-29 | 萨勒普塔医疗公司 | 用于肌肉萎缩症的外显子跳跃寡聚体缀合物 |
| EP3554552B1 (en) | 2016-12-19 | 2022-08-17 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| LT3554553T (lt) | 2016-12-19 | 2022-08-25 | Sarepta Therapeutics, Inc. | Egzoną šalinantys oligomero konjugatai nuo raumenų distrofojos |
| EP3571300A1 (en) | 2017-01-19 | 2019-11-27 | ProQR Therapeutics II B.V. | Oligonucleotide complexes for use in rna editing |
| CN111050806A (zh) | 2017-06-02 | 2020-04-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| WO2018223081A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| AU2018282154B2 (en) | 2017-06-05 | 2022-04-07 | Ptc Therapeutics, Inc. | Compounds for treating huntington's disease |
| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| WO2019005993A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | METHODS OF TREATING HUNTINGTON'S DISEASE |
| CA3067591A1 (en) | 2017-06-28 | 2019-01-03 | Ptc Therapeutics, Inc. | Methods for treating huntington's disease |
| TW201920672A (zh) | 2017-08-08 | 2019-06-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| US11608355B2 (en) | 2017-09-18 | 2023-03-21 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| EA201991450A1 (ru) | 2017-09-22 | 2019-12-30 | Сарепта Терапьютикс, Инк. | Конъюгаты олигомеров для пропуска экзона при мышечной дистрофии |
| JP2020536057A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| EP3687577A1 (en) | 2017-09-28 | 2020-08-05 | Sarepta Therapeutics, Inc. | Combination therapies for treating muscular dystrophy |
| JP2020536060A (ja) | 2017-09-28 | 2020-12-10 | サレプタ セラピューティクス， インコーポレイテッド | 筋ジストロフィーを処置するための併用療法 |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| US12241065B2 (en) | 2018-01-12 | 2025-03-04 | Bristol-Myers Squibb Company | Antisense oligonucleotides targeting alpha-synuclein and uses thereof |
| CN120310791A (zh) | 2018-01-12 | 2025-07-15 | 百时美施贵宝公司 | 靶向α-突触核蛋白的反义寡核苷酸及其用途 |
| AU2019243048A1 (en) | 2018-03-27 | 2020-10-15 | Ptc Therapeutics, Inc. | Compounds for treating Huntington's disease |
| IL277889B2 (en) | 2018-04-12 | 2025-01-01 | Wave Life Sciences Ltd | Oligonucleotide preparations and methods of using them |
| SG11202010131QA (en) | 2018-05-11 | 2020-11-27 | Wave Life Sciences Ltd | Oligonucleotide compositions and methods of use thereof |
| GB201808146D0 (en) | 2018-05-18 | 2018-07-11 | Proqr Therapeutics Ii Bv | Stereospecific Linkages in RNA Editing Oligonucleotides |
| US10758629B2 (en) | 2018-05-29 | 2020-09-01 | Sarepta Therapeutics, Inc. | Exon skipping oligomer conjugates for muscular dystrophy |
| EP3806868A4 (en) | 2018-06-13 | 2022-06-22 | Sarepta Therapeutics, Inc. | EXON SKIPPING OLIGOMERS FOR MUSCULAR DYSTROPHY |
| IL279688B2 (en) | 2018-06-27 | 2025-01-01 | Ptc Therapeutics Inc | Heterocyclic and heteroaryl compounds for the treatment of Huntington's disease |
| BR112020026534A2 (pt) | 2018-06-27 | 2021-03-23 | Ptc Therapeutics, Inc. | Compostos de heteroarila para o tratamento da doença de huntington |
| TW202020153A (zh) | 2018-07-27 | 2020-06-01 | 美商薩羅塔治療公司 | 用於肌肉萎縮症之外顯子跳躍寡聚物 |
| IL321667A (en) | 2018-12-13 | 2025-08-01 | Sarepta Therapeutics Inc | Exon skipping oligomer conjugates for muscular dystrophy |
| BR112021014940A2 (pt) * | 2019-02-01 | 2021-09-28 | Wave Life Sciences Ltd. | Oligonucleotídeo, composição de oligonucleotídeo quiralmente controlada, composição farmacêutica, e método de tratamento, prevenção, atraso de início e/ou diminuição da gravidade de pelo menos um sintoma de doença de huntington |
| US12590115B2 (en) | 2019-03-20 | 2026-03-31 | Wave Life Sciences Ltd. | Technologies useful for oligonucleotide preparation |
| US20220193246A1 (en) | 2019-04-18 | 2022-06-23 | Sarepta Therapeutics, Inc. | Compositions for treating muscular dystrophy |
| AU2020262485A1 (en) * | 2019-04-25 | 2021-11-11 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN114245794B (zh) | 2019-05-13 | 2024-09-13 | Ptc医疗公司 | 用于治疗亨廷顿氏病的化合物 |
| EP4051280A1 (en) | 2019-11-01 | 2022-09-07 | Novartis AG | The use of a splicing modulator for a treatment slowing progression of huntington's disease |
| JP2023526533A (ja) | 2020-05-22 | 2023-06-21 | ウェイブ ライフ サイエンシズ リミテッド | 二本鎖オリゴヌクレオチド組成物及びそれに関連する方法 |
| EP4165205A1 (en) * | 2020-06-15 | 2023-04-19 | CureVac SE | Analysis of nucleic acid mixtures |
| TW202304446A (zh) | 2021-03-29 | 2023-02-01 | 瑞士商諾華公司 | 剪接調節子用於減慢杭丁頓氏舞蹈症進展的治療之用途 |
| EP4396354A4 (en) * | 2021-09-26 | 2025-12-03 | Wave Life Sciences Ltd | OLIGONUCLEOTIDE COMPOSITIONS AND ASSOCIATED PROCESSES |
| JP2024537711A (ja) | 2021-09-30 | 2024-10-16 | サレプタ セラピューティクス， インコーポレイテッド | １つ以上の脱塩基ユニットを有するアンチセンスオリゴヌクレオチド |
| US20250154504A1 (en) | 2022-02-14 | 2025-05-15 | Proqr Therapeutics Ii B.V. | Guide oligonucleotides for nucleic acid editing in the treatment of hypercholesterolemia |
| EP4555085A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Chemically modified oligonucleotides for adar-mediated rna editing |
| EP4555086A1 (en) | 2022-07-15 | 2025-05-21 | ProQR Therapeutics II B.V. | Oligonucleotides for adar-mediated rna editing and use thereof |
| JP2025533454A (ja) | 2022-09-21 | 2025-10-07 | サレプタ セラピューティクス， インコーポレイテッド | Ｄｍｄアンチセンスオリゴヌクレオチド介在性エクソンスキッピング効率 |
| GB202215614D0 (en) | 2022-10-21 | 2022-12-07 | Proqr Therapeutics Ii Bv | Heteroduplex rna editing oligonucleotide complexes |
| WO2024110565A1 (en) | 2022-11-24 | 2024-05-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of hereditary hfe-hemochromatosis |
| GB202218090D0 (en) | 2022-12-01 | 2023-01-18 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of aldehyde dehydrogenase 2 deficiency |
| CN120322556A (zh) | 2022-12-09 | 2025-07-15 | ProQR治疗上市公司Ⅱ | 用于治疗心血管疾病的反义寡核苷酸 |
| GB202300865D0 (en) | 2023-01-20 | 2023-03-08 | Proqr Therapeutics Ii Bv | Delivery of oligonucleotides |
| WO2024175550A1 (en) | 2023-02-20 | 2024-08-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of atherosclerotic cardiovascular disease |
| GB202304363D0 (en) | 2023-03-24 | 2023-05-10 | Proqr Therapeutics Ii Bv | Chemically modified antisense oligonucleotides for use in RNA editing |
| JP2026510408A (ja) | 2023-03-24 | 2026-04-02 | プロキューアール セラピューティクス ツー ベスローテン フェンノートシャップ | 神経障害の処置のためのアンチセンスオリゴヌクレオチド |
| CN121002181A (zh) | 2023-03-27 | 2025-11-21 | ProQR治疗上市公司Ⅱ | 用于治疗肝脏疾病的反义寡核苷酸 |
| TW202442246A (zh) | 2023-04-27 | 2024-11-01 | 美商薩羅塔治療公司 | 用於治療慢性腎病之反義寡聚物 |
| AR132964A1 (es) | 2023-06-16 | 2025-08-13 | Proqr Therapeutics Ii Bv | Oligonucleótidos antisentido para el tratamiento de enfermedades neurodegenerativas |
| WO2025051946A1 (en) | 2023-09-07 | 2025-03-13 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| AU2024362420A1 (en) | 2023-10-18 | 2026-04-23 | Murdoch University | Antisense oligomers for treatment of centronuclear myopathies |
| AU2024379264A1 (en) | 2023-11-16 | 2026-05-14 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of classic galactosemia |
| WO2025132708A1 (en) | 2023-12-20 | 2025-06-26 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of huntington's disease |
| GB202404661D0 (en) | 2024-04-02 | 2024-05-15 | Proqr Therapeutics Ii Bv | Antisense oligoncleotides for the treatment of liver disease |
| WO2025224230A1 (en) | 2024-04-25 | 2025-10-30 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of fatty liver disease |
| GB202410081D0 (en) | 2024-07-11 | 2024-08-28 | Proqr Therapeutics Ii Bv | Antisense oligonucleotides for the treatment of cardiovascular disease |
| WO2026022136A1 (en) | 2024-07-23 | 2026-01-29 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of metabolic disorders |
| WO2026060374A2 (en) | 2024-09-16 | 2026-03-19 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of neurological disorders |
| WO2026068781A1 (en) | 2024-09-30 | 2026-04-02 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of liver disease |
| WO2026093339A1 (en) | 2024-10-29 | 2026-05-07 | Proqr Therapeutics Ii B.V. | Antisense oligonucleotides for the treatment of peroxisome biogenesis disorders in the zellweger spectrum (pbd-zsd) |
Family Cites Families (99)
| Publication number | Priority date | Publication date | Assignee | Title |
|---|---|---|---|---|
| US5512668A (en) * | 1991-03-06 | 1996-04-30 | Polish Academy Of Sciences | Solid phase oligonucleotide synthesis using phospholane intermediates |
| US7015315B1 (en) | 1991-12-24 | 2006-03-21 | Isis Pharmaceuticals, Inc. | Gapped oligonucleotides |
| IL111659A0 (en) | 1993-11-16 | 1995-01-24 | Genta Inc | Synthetic oligomers having chirally pure phosphonate internucleosidyl linkages mixed with non-phosphonate internucleosidyl linkages |
| US6639062B2 (en) | 1997-02-14 | 2003-10-28 | Isis Pharmaceuticals, Inc. | Aminooxy-modified nucleosidic compounds and oligomeric compounds prepared therefrom |
| US6172209B1 (en) | 1997-02-14 | 2001-01-09 | Isis Pharmaceuticals Inc. | Aminooxy-modified oligonucleotides and methods for making same |
| US6867294B1 (en) * | 1998-07-14 | 2005-03-15 | Isis Pharmaceuticals, Inc. | Gapped oligomers having site specific chiral phosphorothioate internucleoside linkages |
| US6242589B1 (en) * | 1998-07-14 | 2001-06-05 | Isis Pharmaceuticals, Inc. | Phosphorothioate oligonucleotides having modified internucleoside linkages |
| US20020082227A1 (en) | 1999-09-30 | 2002-06-27 | Scott Henry | Use of oligonucleotides for inhibition of complement activation |
| DE10019756A1 (de) | 2000-04-20 | 2001-10-25 | Bayer Ag | Verfahren zur Herstellung von superabsorbierenden Polymeren aus Polyacrylnitrilen |
| US20050277133A1 (en) | 2001-05-18 | 2005-12-15 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| JP4348044B2 (ja) | 2002-02-12 | 2009-10-21 | 株式会社キラルジェン | 立体規則性の高いジヌクレオシドホスホロチオエートの製造法 |
| US20050096284A1 (en) | 2002-02-20 | 2005-05-05 | Sirna Therapeutics, Inc. | RNA interference mediated treatment of polyglutamine (polyQ) repeat expansion diseases using short interfering nucleic acid (siNA) |
| US20080274989A1 (en) | 2002-08-05 | 2008-11-06 | University Of Iowa Research Foundation | Rna Interference Suppression of Neurodegenerative Diseases and Methods of Use Thereof |
| US20050042646A1 (en) | 2002-08-05 | 2005-02-24 | Davidson Beverly L. | RNA interference suppresion of neurodegenerative diseases and methods of use thereof |
| US7045306B2 (en) | 2003-04-28 | 2006-05-16 | The General Hospital Corporation | Method for identifying compounds in vitro that modulate the dysregulation of transcription of transcription mediated by mutant huntingtin protein |
| WO2004101787A1 (ja) | 2003-05-14 | 2004-11-25 | Japan Science And Technology Agency | ハンチンチン遺伝子の発現抑制 |
| JP2005089441A (ja) | 2003-08-08 | 2005-04-07 | Toudai Tlo Ltd | 立体規則性の高いリン原子修飾ヌクレオチド類縁体の製造法 |
| WO2005023828A1 (ja) | 2003-09-02 | 2005-03-17 | Takeshi Wada | リボヌクレオチド又はリボヌクレオチド誘導体の製造方法 |
| JP4616175B2 (ja) | 2003-09-02 | 2011-01-19 | 株式会社キラルジェン | ５’−ホスフィチル化モノマーおよびｈ−ホスホネートオリゴヌクレオチド誘導体の製造方法 |
| WO2005027980A1 (en) * | 2003-09-12 | 2005-03-31 | University Of Massachusetts | Rna interference for the treatment of gain-of-function disorders |
| US8680063B2 (en) | 2003-09-12 | 2014-03-25 | University Of Massachusetts | RNA interference for the treatment of gain-of-function disorders |
| JP4945129B2 (ja) | 2004-01-27 | 2012-06-06 | 株式会社キラルジェン | フルオラス担体およびそれを用いたオリゴヌクレオチド誘導体の製造方法 |
| WO2005085272A1 (ja) | 2004-03-05 | 2005-09-15 | Takeshi Wada | ボラノホスフェートモノマーおよびそれを用いたオリゴヌクレオチド誘導体の製造方法 |
| WO2005092909A1 (ja) | 2004-03-25 | 2005-10-06 | Toudai Tlo, Ltd. | 立体規則性の高いリボヌクレオチド類縁体及びデオキシリボヌクレオチド類縁体の製造法 |
| US7902352B2 (en) | 2005-05-06 | 2011-03-08 | Medtronic, Inc. | Isolated nucleic acid duplex for reducing huntington gene expression |
| EP1885854B1 (en) | 2005-05-06 | 2012-10-17 | Medtronic, Inc. | Methods and sequences to suppress primate huntington gene expression |
| US9133517B2 (en) | 2005-06-28 | 2015-09-15 | Medtronics, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin |
| EP2322657B1 (en) | 2005-06-28 | 2014-11-05 | Medtronic, Inc. | Methods and sequences to preferentially suppress expression of mutated huntingtin gene. |
| AU2006305886C1 (en) * | 2005-10-28 | 2011-03-17 | Alnylam Pharmaceuticals, Inc. | Compositions and methods for inhibiting expression of huntingtin gene |
| SI2161038T1 (sl) | 2006-01-26 | 2014-05-30 | Isis Pharmaceuticals, Inc. | Sestavki in njihove uporabe, usmerjeni proti huntingtinu |
| CA2662704A1 (en) | 2006-07-07 | 2008-01-10 | University Of Massachusetts | Rna silencing compositions and methods for the treatment of huntington's disease |
| CA2667055C (en) | 2006-10-18 | 2017-05-09 | Isis Pharmaceuticals, Inc. | Antisense compounds |
| WO2008151833A2 (en) | 2007-06-13 | 2008-12-18 | Hochschule Mannheim | Compounds for the modulation of huntingtin aggregation, methods and means for identifying such compounds |
| EP2014769B1 (en) | 2007-06-18 | 2010-03-31 | Commissariat à l'Energie Atomique | Reversible siRNAa-based silencing of mutated and endogenous wild-type huntingtin gene and its application for the treatment of Huntington's disease |
| CA2711587A1 (en) | 2008-02-04 | 2009-08-13 | Galapagos Nv | Target sequences and methods to identify the same, useful in treatment of neurodegenerative diseases |
| US8679750B2 (en) | 2008-05-09 | 2014-03-25 | The University Of British Columbia | Methods and compositions for the treatment of Huntington'S disease |
| EP2297341A4 (en) * | 2008-05-09 | 2013-01-09 | Univ British Columbia | METHOD AND COMPOSITIONS FOR TREATING MORBUS HUNTINGTON |
| WO2009143391A2 (en) | 2008-05-22 | 2009-11-26 | Isis Pharmaceuticals, Inc | Methods for modulation expression of creb |
| WO2009148605A2 (en) | 2008-06-04 | 2009-12-10 | Isis Pharmaceuticals, Inc. | Methods for treating hypercholesterolemia |
| SG171914A1 (en) | 2008-12-02 | 2011-07-28 | Chiralgen Ltd | Method for the synthesis of phosphorus atom modified nucleic acids |
| WO2010118263A1 (en) * | 2009-04-08 | 2010-10-14 | University Of Massachusetts | Single-nucleotide polymorphism (snp) targeting therapies for the treatment of huntington's disease |
| EP2451461A4 (en) | 2009-07-06 | 2013-05-29 | Ontorii Inc | NOVEL NUCLEIC ACID PRODRUGS AND METHOD OF USE THEREOF |
| WO2011015572A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
| WO2011015573A1 (en) | 2009-08-03 | 2011-02-10 | Galapagos Nv | Molecular targets and compounds, and methods to identify the same, useful in the treatment of neurodegenerative diseases |
| US8927553B2 (en) | 2009-08-10 | 2015-01-06 | Daljit Singh Dhanoa | Deuterium-enriched alkyl sulfonamides and uses thereof |
| RU2562861C2 (ru) | 2009-09-11 | 2015-09-10 | Айсис Фармасьютикалс, Инк. | Модуляция экспрессии гентингтина |
| CN102574888A (zh) | 2009-09-16 | 2012-07-11 | 株式会社启拉坚 | 用于rna及其衍生物的合成的新型保护基 |
| DK2519632T3 (en) | 2009-12-31 | 2018-07-23 | Curna Inc | TREATMENT OF INSULIN RECEPTOR SUBSTRATE 2- (IRS2) RELATED DISEASES BY INHIBITION OF NATURAL ANTISENSE TRANSCRIPTION TO IRS2 AND TRANSCRIPTION FACTOR E3 (TFE3) |
| EP2534262B1 (en) | 2010-02-08 | 2016-12-14 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| EP3561060A1 (en) * | 2010-02-08 | 2019-10-30 | Ionis Pharmaceuticals, Inc. | Selective reduction of allelic variants |
| WO2011108682A1 (ja) | 2010-03-05 | 2011-09-09 | 国立大学法人 東京大学 | リボヌクレオシドホスホロチオエートの製造方法 |
| JP2011184318A (ja) | 2010-03-05 | 2011-09-22 | Univ Of Tokyo | リボヌクレシドｈ−ボラノホスホネート |
| DK2620428T3 (da) | 2010-09-24 | 2019-07-01 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| AU2011325956B2 (en) | 2010-11-12 | 2016-07-14 | The General Hospital Corporation | Polycomb-associated non-coding RNAs |
| EP2647644B1 (en) | 2010-11-30 | 2020-09-09 | Wave Life Sciences Japan, Inc. | 2'-o-modified rna |
| EP3067421B1 (en) | 2011-02-08 | 2018-10-10 | Ionis Pharmaceuticals, Inc. | Oligomeric compounds comprising bicyclic nucleotides and uses thereof |
| WO2012109667A1 (en) | 2011-02-12 | 2012-08-16 | University Of Iowa Research Foundation | Therapeutic compounds |
| US9605019B2 (en) | 2011-07-19 | 2017-03-28 | Wave Life Sciences Ltd. | Methods for the synthesis of functionalized nucleic acids |
| US10202599B2 (en) | 2011-08-11 | 2019-02-12 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| WO2013082548A1 (en) | 2011-11-30 | 2013-06-06 | Sarepta Therapeutics, Inc. | Oligonucleotides for treating expanded repeat diseases |
| CN112007045B (zh) * | 2012-07-13 | 2026-01-06 | 波涛生命科学有限公司 | 手性控制 |
| DK2872485T3 (da) | 2012-07-13 | 2021-03-08 | Wave Life Sciences Ltd | Asymmetrisk hjælpegruppe |
| JP6246121B2 (ja) | 2012-07-13 | 2017-12-13 | 株式会社新日本科学 | キラル核酸アジュバント |
| US20150275208A1 (en) | 2012-10-12 | 2015-10-01 | Isis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| US10260069B2 (en) | 2013-02-04 | 2019-04-16 | Ionis Pharmaceuticals, Inc. | Selective antisense compounds and uses thereof |
| CA2921162A1 (en) | 2013-05-01 | 2014-11-06 | Ionis Pharmaceuticals, Inc. | Conjugated antisense compounds and their use |
| KR20160067219A (ko) | 2013-10-03 | 2016-06-13 | 모더나 세라퓨틱스, 인코포레이티드 | 저밀도 지단백질 수용체를 암호화하는 폴리뉴클레오타이드 |
| AU2014346424B2 (en) | 2013-11-11 | 2020-09-17 | Sangamo Therapeutics, Inc. | Methods and compositions for treating Huntington's Disease |
| US10322173B2 (en) | 2014-01-15 | 2019-06-18 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having anti-allergic activity, and anti-allergic agent |
| EP3095461A4 (en) | 2014-01-15 | 2017-08-23 | Shin Nippon Biomedical Laboratories, Ltd. | Chiral nucleic acid adjuvant having immunity induction activity, and immunity induction activator |
| WO2015108048A1 (ja) | 2014-01-15 | 2015-07-23 | 株式会社新日本科学 | 抗腫瘍作用を有するキラル核酸アジュバンド及び抗腫瘍剤 |
| KR102423317B1 (ko) | 2014-01-16 | 2022-07-22 | 웨이브 라이프 사이언시스 리미티드 | 키랄 디자인 |
| WO2015168172A1 (en) | 2014-04-28 | 2015-11-05 | Isis Pharmaceuticals, Inc. | Linkage modified oligomeric compounds |
| CN106413760B (zh) | 2014-05-08 | 2020-01-14 | 桑格摩生物科学股份有限公司 | 用于治疗亨廷顿病的方法和组合物 |
| ES2759317T3 (es) | 2014-05-20 | 2020-05-08 | Univ Iowa Res Found | Compuestos terapéuticos para la enfermedad de Huntington |
| US20160017327A1 (en) | 2014-07-11 | 2016-01-21 | The Johns Hopkins University | Phosphorodiamidate morpholino oligomers (pmos) and their use in suppression of mutant huntingtin expression and attenuation of neurotoxicity |
| EP3169693B1 (en) | 2014-07-16 | 2022-03-09 | ModernaTX, Inc. | Chimeric polynucleotides |
| RU2708237C2 (ru) * | 2014-08-22 | 2019-12-05 | Общество с ограниченной ответственностью "НооГен" | Модифицированные олигонуклеотиды и способ их получения |
| JP2017536119A (ja) | 2014-11-19 | 2017-12-07 | ロシュ イノベーション センター コペンハーゲン エーエス | キラルホスホロチオエート連結を含むｌｎａギャップマーオリゴヌクレオチド |
| EP3237618B1 (en) | 2014-12-24 | 2019-05-22 | uniQure IP B.V. | Rnai induced huntingtin gene suppression |
| EP3253871A1 (en) | 2015-02-04 | 2017-12-13 | Bristol-Myers Squibb Company | Lna oligonucleotides with alternating flanks |
| US10450563B2 (en) | 2015-02-10 | 2019-10-22 | Genzyme Corporation | Variant RNAi |
| JP7009356B2 (ja) | 2015-04-03 | 2022-01-25 | ユニバーシティ・オブ・マサチューセッツ | ハンチンチンｍＲＮＡをターゲティングするオリゴヌクレオチド化合物 |
| WO2017015575A1 (en) | 2015-07-22 | 2017-01-26 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
| EP3359523A4 (en) | 2015-10-09 | 2019-07-24 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHOD THEREFOR |
| AU2017234150B2 (en) | 2016-03-13 | 2021-09-16 | Wave Life Sciences Ltd. | Compositions and methods for phosphoramidite and oligonucleotide synthesis |
| US20190249173A1 (en) | 2016-05-04 | 2019-08-15 | Chandra Vargeese | Methods and compositions of biologically active agents |
| JP2019516680A (ja) | 2016-05-04 | 2019-06-20 | ウェイブ ライフ サイエンシズ リミテッドＷａｖｅ Ｌｉｆｅ Ｓｃｉｅｎｃｅｓ Ｌｔｄ． | オリゴヌクレオチド組成物およびその方法 |
| US11013757B2 (en) | 2016-06-03 | 2021-05-25 | Wave Life Sciences Ltd. | Oligonucleotides, compositions and methods thereof |
| WO2018022473A1 (en) | 2016-07-25 | 2018-02-01 | Wave Life Sciences Ltd. | Phasing |
| JP7296882B2 (ja) | 2016-11-23 | 2023-06-23 | ウェイブ ライフ サイエンシズ リミテッド | ホスホラミダイト及びオリゴヌクレオチド合成のための組成物及び方法 |
| EP3630788A4 (en) | 2017-06-02 | 2021-04-28 | Wave Life Sciences Ltd. | OLIGONUCLEOTIDE COMPOSITIONS AND METHODS FOR THEIR USE |
| WO2018223081A1 (en) | 2017-06-02 | 2018-12-06 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods of use thereof |
| CN111050806A (zh) | 2017-06-02 | 2020-04-21 | 波涛生命科学有限公司 | 寡核苷酸组合物及其使用方法 |
| WO2018237194A1 (en) | 2017-06-21 | 2018-12-27 | Wave Life Sciences Ltd. | Compounds, compositions and methods for synthesis |
| EP3646836B1 (en) | 2017-06-26 | 2021-10-06 | Yamaha Hatsudoki Kabushiki Kaisha | Power assist wheelchair, power assist unit for wheelchair, control device for power assist wheelchair, control method for power assist wheelchair, and program |
| TW201920672A (zh) | 2017-08-08 | 2019-06-01 | 新加坡商波濤生命科學有限公司 | 寡核苷酸組合物及其使用方法 |
| US11608355B2 (en) | 2017-09-18 | 2023-03-21 | Wave Life Sciences Ltd. | Technologies for oligonucleotide preparation |
| US11596646B2 (en) | 2017-10-12 | 2023-03-07 | Wave Life Sciences Ltd. | Oligonucleotide compositions and methods thereof |
-
2017
- 2017-05-03 JP JP2018557087A patent/JP2019516680A/ja active Pending
- 2017-05-03 US US16/098,836 patent/US10724035B2/en not_active Expired - Fee Related
- 2017-05-03 WO PCT/US2017/030753 patent/WO2017192664A1/en not_active Ceased
- 2017-05-03 MA MA045270A patent/MA45270A/fr unknown
- 2017-05-03 CN CN201780038913.4A patent/CN109477108A/zh active Pending
- 2017-05-03 EP EP17793215.9A patent/EP3452596A4/en not_active Withdrawn
-
2020
- 2020-05-07 US US16/869,126 patent/US20220162598A1/en not_active Abandoned
Similar Documents
| Publication | Publication Date | Title |
|---|---|---|
| JP2022531539A5 (enExample) | ||
| JP2021522803A5 (enExample) | ||
| JP2018502956A5 (enExample) | ||
| JP2017532038A5 (enExample) | ||
| JP2018525357A5 (enExample) | ||
| JP2019504112A5 (enExample) | ||
| IL316808A (en) | Modified double-stranded RNA materials and their uses | |
| JP2020530442A5 (enExample) | ||
| JP2021511312A5 (enExample) | ||
| TW201540724A (zh) | 反義核酸 | |
| CN114025848A (zh) | 用于调节剪接和翻译的方法和组合物 | |
| JP2021530541A5 (enExample) | ||
| JP2022533358A5 (enExample) | ||
| JP2017511377A5 (enExample) | ||
| JP2020522567A5 (enExample) | ||
| JP2016516773A5 (enExample) | ||
| CN112996913A (zh) | 寡聚核酸分子及其应用 | |
| US20190071670A1 (en) | Methods Of Treatment For Alpha-1 Antitrypsin Deficiency | |
| JP2011515357A5 (enExample) | ||
| JP2017503753A5 (enExample) | ||
| JP2013537404A5 (enExample) | ||
| JP2015502973A5 (enExample) | ||
| WO2010039187A3 (en) | Fused diimidazodiazepine compounds and methods of use and manufacture thereof | |
| TW202405171A (zh) | 血管收縮素原調節組合物及其使用方法 | |
| TW200906842A (en) | Salts of isophosphoramide mustard and analogs thereof |